Study on Venetoclax and ASTX727 for Untreated High-Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia

Sponsor: M.D. Anderson Cancer Center

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT04655755

Provider View

This clinical trial is testing a new combination of drugs, venetoclax and ASTX727, to find the best dose and understand the side effects in patients with high-risk myelodysplastic syndrome or chronic myelomonocytic leukemia who have not received any prior treatment.

Patient Parameters

Program Overview

This study is exploring the effects of combining venetoclax with ASTX727 in patients who have high-risk myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) and have not been treated before. The trial aims to find the safest and most effective dose of this combination. Venetoclax, cedazuridine, and decitabine (ASTX727) are chemotherapy drugs that work by stopping cancer cells from growing, dividing, or spreading.

Description

The main goal of this study is to determine how safe and tolerable the combination of venetoclax and ASTX727 is for patients with untreated high-risk MDS or CMML. The study will also look at how well the treatment works in reducing cancer cells in the bone marrow.

Secondary goals include:

  • Checking the rate of complete remission, where no cancer cells are detectable.
  • Observing improvements in blood cell counts.
  • Seeing if patients can become independent of blood transfusions.
  • Monitoring changes in genetic markers and cancer cell responses.
  • Measuring how long it takes for the disease to progress to acute myeloid leukemia (AML).
  • Evaluating overall survival and time without disease progression.

The study is conducted in two phases: Phase I focuses on finding the best dose, and Phase II evaluates the treatment's effectiveness. Patients will take venetoclax by mouth once daily for 14 days and ASTX727 for 5 days in a 28-day cycle, as long as the treatment is effective and side effects are manageable. After completing the treatment, patients will have follow-up visits every 3-6 months for up to 5 years.

Eligibility Criteria

Inclusion Criteria

  • Have not received any prior treatment for high-risk myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML).
  • Have a specific level of cancer cells in the bone marrow.
  • Meet certain blood test requirements for liver and kidney function.
  • Be 18 years or older.
  • Women must not be pregnant and must agree to use birth control during the study and for 3 months after.
  • Men must agree to use birth control during the study and for 3 months after.
  • Provide written informed consent to participate in the study.

Exclusion Criteria

  • Have received any previous treatment with BCL2 inhibitors.
  • Have low-risk MDS according to specific medical criteria.
  • Have HIV, hepatitis B, or hepatitis C infections unless under control.
  • Have taken certain medications or foods that interact with the study drugs within a specified time before starting the study.
  • Have significant heart, lung, kidney, liver, or other medical conditions that could interfere with the study.
  • Have a history of other cancers within the last 2 years, except for certain skin cancers or other cancers treated with curative intent.
  • Have a high white blood cell count unless controlled with medication.
  • Have received a live vaccine within 4 weeks before starting the study.
  • Women who are pregnant or have a positive pregnancy test.

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